News BioMarin halts Voxzogo trials due to safety signal BioMarin has suspended dosing in phase 2 trials of its achondroplasia therapy Voxzogo aimed at broadening the drug's label.
News BridgeBio gains on phase 3 dwarfism data Shares in BridgeBio rose after it revealed data from a trial of infigratinib in achondroplasia, raising the prospect of the first oral treatment.
News BioMarin ends year with $4.8bn play for Amicus BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.
News NHS cleared to use AZ's Fasenra for rare autoimmune disease NICE backs NHS use of an AstraZeneca drug for one rare disease, but says it can't recommend a BioMarin therapy for another.
News BioMarin starts rebuilding after its downsizing spree BioMarin has made its first M&A play under James Sabry, buying Inozyme to flesh out its enzyme replacement therapies (ERTs) for rare diseases.
News Spruce Bio sows the seeds of its new future Spruce has bounced back from the failure of its lead drug last year, thanks to a licensing deal with BioMarin for a late-stage Sanfilippo drug.
Patients LSX2026: On promise and patience, with Hans Schambye At LSX World Congress 2026 in Lisbon, Portugal, web editor Nicole Raleigh spoke with Hans Schambye, CEO of BOOST Pharma.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
